Ongoing Clinical Trials for Acute Leukaemia
Currently, 6 clinical trials are investigating new treatment approaches for acute leukaemia, including studies on targeted therapies, infection prevention strategies, stem cell transplantation methods, and fertility preservation during treatment. These trials are being conducted across multiple European countries and offer options for both adults and children with various forms of the disease.
Clinical trial locations
- Belgium
- France
- Germany
- Italy
- Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children
- Study Comparing Pevonedistat, Venetoclax, and Azacitidine with Venetoclax and Azacitidine in Adults with Acute Myeloid Leukemia Unfit for Intensive Chemotherapy
- Study on the Safety and Tolerability of SNDX-5613 for Patients with Relapsed or Refractory Acute Leukemias
- Lithuania
- Netherlands
- Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children
- Study of UM171-expanded cord blood transplantation for patients with high-risk and very high-risk acute leukemia or myelodysplasia
- Study on the Safety and Tolerability of SNDX-5613 for Patients with Relapsed or Refractory Acute Leukemias
- Poland
- Spain
- Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children
- Study Comparing Fosfomycin and Ciprofloxacin for Preventing Fever in Patients with Acute Leukemia After Chemotherapy or Stem Cell Transplant
- Study on the Safety and Tolerability of SNDX-5613 for Patients with Relapsed or Refractory Acute Leukemias
- Sweden
Study on Ponatinib for Treating Recurrent or Refractory Leukemias and Solid Tumors in Children
This trial is testing Ponatinib, a kinase inhibitor medication, in children with blood cancers or solid tumors that have returned after treatment or have not responded to previous treatments.
Main inclusion criteria: Children between 1 and 18 years old with confirmed leukemias, lymphomas, or solid tumors that have not responded to standard treatments or for whom standard treatments are not suitable. Participants must have recovered from previous treatment side effects and have a certain level of physical ability to carry out daily activities. Parents or legal guardians must provide consent.
Main exclusion criteria: Children with different types of cancer than those being studied, those not within the specified age range, those unable to take oral medication, those with other serious health conditions that might interfere with treatment, pregnant or breastfeeding patients, and those participating in other clinical trials simultaneously.
Study focus: The trial has two phases. The first phase aims to find the best and safest dose of Ponatinib for children with these cancers. The second phase evaluates how well Ponatinib works, particularly in children with Chronic Myeloid Leukemia and other selected cancers. Researchers will monitor participants’ health closely, assess treatment response through medical tests and imaging studies, and track any side effects.
Investigational drug: Ponatinib is taken orally once daily in tablet or capsule form. It works by blocking certain proteins that help cancer cells grow.
Study Comparing Fosfomycin and Ciprofloxacin for Preventing Fever in Patients with Acute Leukemia After Chemotherapy or Stem Cell Transplant
This Spanish trial compares two antibiotics, fosfomycin and ciprofloxacin, to determine which works better in preventing febrile neutropenia—a serious condition where patients develop fever alongside dangerously low levels of infection-fighting white blood cells.
Main inclusion criteria: Adults aged 18 or older with newly diagnosed acute leukemia receiving their first chemotherapy treatment or awaiting a first stem cell transplant. Participants must expect to have low neutrophil levels lasting at least seven days. Those with expected moderate decreases must have additional risk factors such as severe mouth sores, age 65 or older, multiple other health conditions, low albumin levels, or high doses of certain chemotherapy drugs. Adequate liver and kidney function is required.
Main exclusion criteria: Patients not at high risk of infection after chemotherapy, those without acute leukemia, those not undergoing intensive chemotherapy or stem cell transplantation, patients outside the specified age range, and vulnerable populations requiring special protection.
Study focus: The study randomly assigns participants to receive either fosfomycin or ciprofloxacin, both taken orally. Researchers monitor participants throughout their treatment to assess how effectively each antibiotic prevents infections and to ensure patient safety during the vulnerable period when white blood cell counts are low.
Investigational drugs: Both fosfomycin and ciprofloxacin are antibiotics that work by stopping bacterial growth, helping to prevent serious infections in patients with weakened immune systems.
Study of UM171-expanded cord blood transplantation for patients with high-risk and very high-risk acute leukemia or myelodysplasia
This Netherlands-based trial investigates a specialized cord blood transplantation method for patients with high-risk blood disorders who require stem cell transplantation.
Main inclusion criteria: Adults between 18 and 70 years old diagnosed with high or very high-risk blood cancer. Two matching cord blood units must be available. Participants must have a Karnofsky score of 70 or higher (indicating reasonable ability to perform daily activities), adequate heart function with ejection fraction of at least 40%, lung function at least 50% of normal, acceptable liver function tests, and adequate kidney function. Overall health assessment scores must meet specific thresholds depending on disease status and age.
Main exclusion criteria: Active uncontrolled infections, severe heart, liver, or kidney problems, pregnant or breastfeeding women, previous stem cell transplantation, active autoimmune diseases, HIV positive status, active hepatitis B or C infection, mental conditions interfering with study procedures, recent participation in other trials, known allergies to study medications, uncontrolled high blood pressure, and active secondary cancer.
Study focus: The treatment involves cord blood cells that have been specially processed using ECT-001 technology to increase the number of blood-forming stem cells. Patients receive these processed cells through intravenous infusion. Researchers monitor how well the new cells establish themselves in the patient’s body and track recovery for up to two years, assessing blood count recovery and checking for immune system complications.
Investigational drug: ECT-001-CB consists of umbilical cord blood cells treated with UM171, a molecule that expands the number of blood-forming stem cells, potentially improving transplant success rates.
Study on Triptorelin for Fertility Protection in Young Women and Teenagers Undergoing Chemotherapy for Breast Cancer, Leukemia, Lymphomas, and Sarcomas
This Swedish trial examines whether GnRHa (Gonadotropin-Releasing Hormone agonist) can help preserve fertility in young women and teenagers undergoing chemotherapy for various cancers, including acute leukemia.
Main inclusion criteria: Females aged 14-42 years at cancer diagnosis with breast cancer, acute leukemia, lymphomas (Hodgkin or non-Hodgkin), or sarcomas (osteosarcoma, soft tissue, or Ewing sarcoma) confirmed by tissue testing and scheduled for disease-specific chemotherapy. Participants must have started menstrual periods, have performance status of 0-1 (able to carry out light work), and have adequate bone marrow, kidney, liver, and heart function without uncontrolled medical or psychiatric disorders.
Main exclusion criteria: Males, patients without the specified cancer types, those outside the age range, and vulnerable populations.
Study focus: Participants are randomly assigned to receive either GnRHa treatment (Pamorelin at 3.75 mg or 11.25 mg doses) or placebo through intramuscular injection. The study monitors ovarian reserve—a measure of remaining egg supply—by measuring Anti-Müllerian Hormone levels at various points after chemotherapy ends. Follow-up continues for up to five years, including ultrasounds, blood tests, and quality of life assessments to determine if GnRHa helps preserve fertility during cancer treatment.
Investigational drug: GnRHa works by temporarily putting the ovaries to rest, potentially shielding them from chemotherapy’s harmful effects and preserving future fertility.
Study Comparing Pevonedistat, Venetoclax, and Azacitidine with Venetoclax and Azacitidine in Adults with Acute Myeloid Leukemia Unfit for Intensive Chemotherapy
This trial, conducted in Poland and Italy, compares two treatment combinations for adults with newly diagnosed Acute Myeloid Leukemia who cannot undergo intensive chemotherapy.
Main inclusion criteria: Adults aged 18 or older with newly diagnosed AML confirmed by a doctor. Patients can have primary AML or secondary AML (developing after other blood disorders or treatments). Participants must be unfit for intensive chemotherapy due to being 75 years or older, or aged 18-74 with conditions such as poor physical health status, severe heart or lung problems, reduced kidney function, or liver problems. Laboratory tests must show total bilirubin, liver enzymes, kidney function, and albumin levels within acceptable ranges. White blood cell count must be less than 25 x 10⁹/L.
Main exclusion criteria: Patients with cancers other than AML, those fit for intensive chemotherapy, previous treatment with the study drugs, uncontrolled or severe heart disease, active uncontrolled infections, pregnant or breastfeeding women, history of severe allergic reactions to study drugs, recent receipt of other investigational drugs, and other serious medical conditions that could interfere with the study.
Study focus: One group receives a three-drug combination of Pevonedistat (given intravenously), Venetoclax (oral tablets), and Azacitidine (intravenous or subcutaneous). The comparison group receives Venetoclax and Azacitidine only. Treatment occurs in 28-day cycles with regular health assessments including blood tests and physical exams. The study evaluates whether the three-drug combination is more effective than the two-drug combination in terms of how long patients live and how the disease responds to treatment.
Investigational drugs: Pevonedistat blocks specific proteins in cancer cells to stop growth. Venetoclax targets and blocks a protein helping cancer cells survive, leading to their death. Azacitidine interferes with cancer cell growth by incorporating into DNA and disrupting growth processes.
Study on the Safety and Tolerability of SNDX-5613 for Patients with Relapsed or Refractory Acute Leukemias
This trial across six European countries investigates Revumenib (SNDX-5613) for patients with blood cancers that have returned after treatment or have not responded to previous treatments.
Main inclusion criteria: Patients must have leukemia that has relapsed or proven refractory to treatment, including cases with specific genetic changes like KMT2A rearrangement or NPM1 mutation. Previous immunotherapy must be completed at least 42 days ago, certain cell therapies at least 21 days ago, and other anti-leukemia treatments at least 14 days ago. Blood cell growth factor treatments must be finished at least 7 days ago for short-acting types and 14 days ago for long-acting types. Patients must be at least 6 months old (with weight requirements for certain treatment combinations), have adequate physical ability for daily activities, and have previous treatment side effects improved to mild levels. Radiation treatments must be completed at least 60 days ago for extensive radiation or 14 days for local radiation, and stem cell transplants at least 60 days ago.
Main exclusion criteria: Patients without relapsed or refractory leukemia, those who haven’t tried other treatments, patients outside the age range, those unable to safely tolerate the medication or follow study procedures, patients with interfering medical conditions, pregnant or breastfeeding patients, those in other concurrent trials, patients with allergic reactions to similar medications, and those who haven’t provided consent.
Study focus: The trial has two phases. Phase 1 focuses on finding the right dose by testing various doses of Revumenib (available as oral solution, capsule, or tablet) while monitoring safety and tolerability. Phase 2 evaluates the drug’s safety over a longer period and its ability to help patients achieve remission. The study specifically targets patients with MLL/KMT2A gene rearrangement or NPM1 mutation, as these genetic changes affect how leukemia behaves and responds to treatment.
Investigational drug: SNDX-5613 (Revumenib) is a targeted therapy taken orally that works by inhibiting a protein involved in cancer cell growth and survival, potentially stopping disease progression.
Summary
The six ongoing clinical trials for acute leukaemia reflect diverse approaches to managing this challenging disease. The trials span treatment options for both children and adults, addressing different stages and circumstances of the disease.
Geographically, the trials show concentration in certain countries, with Italy participating in four trials, followed by the Netherlands, Spain, and France with three trials each. Several trials are multinational, particularly those investigating targeted therapies like Ponatinib and SNDX-5613, which operate across multiple European countries.
The research focuses on several key areas: targeted therapies blocking specific proteins involved in cancer cell growth (Ponatinib, Pevonedistat, Venetoclax, SNDX-5613), infection prevention during vulnerable treatment periods (fosfomycin and ciprofloxacin comparison), innovative stem cell transplantation methods using expanded cord blood cells, and supportive care including fertility preservation during chemotherapy.
Notably, several trials specifically address patients for whom intensive chemotherapy is not suitable, recognizing the need for effective treatment options for older adults or those with other health conditions. The trials also include important quality-of-life considerations, such as the fertility preservation study for young women and teenagers facing cancer treatment.
These studies represent important steps toward improving outcomes and quality of life for patients with acute leukaemia across different age groups and disease circumstances.
